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Duchenne Muscular Dystrophy


Kevin Brennan (Cardiff, West): I hope that this important debate will raise awareness of the serious medical condition of Duchenne muscular dystrophy.

Duchenne muscular dystrophy is named after Dr. Duchenne de Boulogne, who worked in Paris in the mid-19th century and was one of the first people to study muscular dystrophy. The problem is known to result from a gene defect in a single important fibre in muscle fibres called dystrophin. Without dystrophin to strengthen them, the muscle cells become weaker and eventually waste away.

DMD is the most common, most progressive and most deadly type of muscular dystrophy. It is an X chromosome-linked disease, and as such predominantly affects boys; worldwide, it affects one in 3,500 boys. DMD is usually diagnosed when a child reaches two to three years of age and has not reached developmental milestones such as standing and walking. As such children progress through childhood they are often prone to falls and are eventually unable to walk—they usually need a wheelchair by the age of 11. Through their teen years, such children will become progressively disabled as their muscles seriously weaken and waste away. In the final years of their life, 24-hour care will be required including night-time ventilation and feeding support. A young adult with DMD is unlikely to live beyond their 21st birthday, and death is usually caused by heart or lung failure as the muscles supporting the vital organs finally become so weak that they are unable to maintain life.

DMD is sometimes called an equal opportunity condition. Although many cases of DMD are hereditary, an increasing number of cases seem to be caused by spontaneous mutations. As such, the condition could cast its shadow over anybody of any background or nationality. In more than one sense, it is a case of genetic roulette.

I was first alerted to DMD earlier this year when two of my constituents, Nick Catlin and Janet Hoskin, approached me for help. Nick and Janet have a two-year-old son called Saul, and, without breaching any parliamentary rules, I can say that I know that they are taking a close interest in today's proceedings. Saul was diagnosed as having DMD at the unusually young age of five months following a routine blood test for liver function. Saul is Nick and Janet's first child, and I am sure that you will allow me to take this opportunity, Mr. Griffiths, to congratulate them on the birth of their second child, a girl called Amelia, who was born last week. I wish them all the best for the future.

Saul was Nick and Janet's first child, and receiving and accepting Saul's diagnosis was devastating for the family. Writing in February, Nick told me:

In the same letter, Janet added that they wanted the Government

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At present, Saul is a healthy, busy toddler more concerned with cars and diggers than health matters. It is very hard for anyone to believe that such a vibrant little boy will be condemned to such a short life in which he will never be able to fulfil his potential. The plight of their son and the intense desire to find a cure for the condition led Nick and Janet to form their own charity, Parent Project UK, in February 2002. One of the main spurs behind the establishment of PPUK was the passing of the Muscular Dystrophy Community Assistance, Research and Education Act through the United States Congress on 21 December 2001, which effectively ring-fenced millions of dollars for research into DMD. The main aims of PPUK are to raise awareness of DMD, to raise money for research and, like any good lobbying group, to put pressure on the Government to release funds to find a cure. At present, only a limited amount of Government money is earmarked for research into this specific area and scientists are forced to compete for funds with research into other serious illnesses.

Since the charity was founded, Nick, Janet and the other helpers of PPUK have worked long and hard to raise awareness of Duchenne muscular dystrophy and campaign for funding for new research. Nick and Janet were lucky enough to receive a start-up grant of £5,000 from the National Lottery and on Saturday 27 July this year members of the Royal Monmouthshire Royal Engineers field support squadron took part in a relay organised by PPUK between Swansea and Cardiff. To date, that run has raised another £2,000. I am also pleased and delighted to say that PPUK has successfully bid for a grant of £48,950 from the community fund, which, over the coming year, will cover the cost of a project co-ordinator and the general running costs of the new charity.

As a relative newcomer on the scene, PPUK is following in the footsteps of two other charitable and lobbying organisations—the Muscular Dystrophy Campaign and the Duchenne Family Support Group. The Muscular Dystrophy Campaign is a national charity focusing on all the muscular dystrophies and allied disorders. It has pioneered the search for treatments and cures for more than 40 years and aims to provide medical and emotional support for sufferers and their families.

The Duchenne Family Support Group is a national charity. It was founded in 1987 and is run by and for families affected by DMD. The group strives to bring families together for mutual support, sharing of information and social activities. In recent months, the three charities have pooled their resources to co-ordinate a nationwide campaign called "The Right to Survive". The campaign aims to increase funding for research into finding a cure for Duchenne muscular dystrophy to give sufferers the right to survive into adulthood. Those charities are requesting that money be dedicated to research into treatments for this disease. Children born now with Duchenne muscular dystrophy could have the chance of a longer life. The UN convention on the rights of the child was adopted by the United Kingdom in 1991 and states that the Government are committed to children's right to life and to survival and development. The right to survive campaign calls in that pledge to give boys with Duchenne muscular dystrophy the right to survive into

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adulthood. The campaign has operated a three-pronged approach in its attempt to raise awareness. Earlier this year, on 20 May, I tabled early-day motion 1341, which has so far attracted 85 signatures from hon. Members of all parties in the House. I hope that as a result of this debate that figure will rise. The right to survive campaign has also been gaining pledges of support from the public. I shall be taking members of that campaign to No. 10 Downing street this afternoon to hand in petitions of support.

Tonight my hon. Friend the Member for Norwich, North (Dr. Gibson) and I will host a briefing in Committee Room 11, at which Members of both Houses of Parliament will be given the opportunity to hear some of the experiences of DMD sufferers' families and to discuss the issues surrounding DMD with campaigns from all three charities. I urge Members of this and the other place to spare time this evening to come to the briefing, for however short a time, and to find out more about Duchenne muscular dystrophy first hand.

The other main aim of the campaign is to try to persuade the Government, the Medical Research Council or anyone else to release more funding for research into a cure for Duchenne muscular dystrophy. At present, the Muscular Dystrophy Campaign is the only voluntary organisation in the United Kingdom funding scientific research into potential cures and treatments for all types of muscular dystrophy and related disorders. The charity supports a programme of around 30 research projects in the UK and Europe with an investment of £1.5 million in 2002. Those projects are working to characterise the genes and proteins responsible for different types of muscular dystrophy as well as investigating techniques for potential treatments and cures. Results from the projects are shared among the international science community to ensure that knowledge about the range of initiatives under investigation is pooled effectively.

The main agency through which the Government support medical and clinical research is the Medical Research Council. The MRC is an independent body and it receives its grant in aid from the Office of Science and Technology. As such, it operates under the auspices of the Department for Trade and Industry. MRC spending on muscular dystrophy research for 2001–02 is estimated at £950,000, or 0.24 per cent. of its £394 million gross expenditure. That proportion has been consistent over the past six years. In addition, the Department of Health funds the NHS research and development budget.

At current levels, the voluntary sector funds a third more research than the Government. Charities rightly find that unacceptable, and they are calling for increased ring-fenced funding for Duchenne muscular dystrophy. What plans, if any, do the Government have in that regard? I hope that my hon. Friend the Minister will say something about that.

Any change in Government funding will require a shift in strategy from the MRC. There is currently limited funding for projects associated with so-called rare conditions, and grant applications for research into muscular dystrophy often receive the highest technical ranking from the MRC only to be turned down on funding grounds. As part of the ongoing campaign, PPUK, the Muscular Dystrophy Campaign and the

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Duchenne Family Support Group intend to meet the MRC to discuss ways of overcoming the funding issue and to prepare the ground for potential clinical trials in the United Kingdom.

Dr. Ian Gibson (Norwich, North): My hon. Friend may be aware that the MRC now has a strategy of denying money to alpha-rated projects of great international prominence in the fields of brain function, leukaemia and asthma. It recently adopted a policy of funding only massive projects involving five or six groups. The chances of its funding what it may see as small problems, such as muscular dystrophy, seem remoter than ever. There must be pressure on the MRC to examine its funding strategy for research into rarer and more prominent diseases.

Kevin Brennan : I am grateful to my hon. Friend for that; he obviously speaks with great expertise and knowledge. He emphasises my point that it is vital for the MRC to reconsider its approach.

Another aspect of DMD that I want to mention is the method of genetic research employed. Dystrophin's role in the cell is not fully understood, and scientists are trying to determine it. One of its roles is thought to be structural, due to its position in the muscle cell membrane where it forms part of the dystrophin-glycoprotein complex, or DGC. It forms a link between the outside and the inside of the muscle membrane. Without it, the membrane becomes weak and breaks down, and the muscle cell dies.

The main body of research aims to pinpoint why dystrophin is absent in certain cases, and there are three main avenues of research: muscle cell transfer, gene therapy and more general pharmacological research. Muscle cell transfer utilises genetically modified self cells, and research using experimental models has shown that skin cells that are grown outside the body in a liquid medium that previously contained muscle cells may convert into muscle cells. Research is under way to find the factor responsible for that conversion—a reaction referred to as galectin 1—and to gain a better understanding of the type of cell that is capable of it. Should there be therapeutic potential, skin cells from a person with DMD could be removed and a correct version of the dystrophin gene could be inserted. The cells could then be grown and reintroduced into the person, where they would help to repopulate the muscle and form functional muscle tissue.

Gene therapy—or gene repair technology, to use the more technically accurate description—examines ways of repairing the gene in situ or outside the body and then putting the cells back. This new technology can use repair mechanisms that cells normally use to target and correct specific mutations. It does not involve introducing a new gene into cells, but repairs the existing, faulty gene. It may benefit 15 to 30 per cent. of individuals with DMD.

Both those types of research depend on stem cell research, and it is important that we applaud the Government's decision to continue it. It is a notable, if gloomy, irony that the United States has ring-fenced money for DMD research but at the same time called a

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moratorium on stem cell research, which may have the most potential for finding a cure. It would be a tragedy if progress towards a cure were held up due to lack of resolve in the US Administration when funds were available, which is why it is important that funding be available in the United Kingdom for stem cell research.

Pharmacological approaches to DMD treatment could bring a more traditional range of approaches to alleviating some of the symptoms, if not to finding an eventual cure. Perhaps a combination of the three approaches might be the answer.

Every hon. Member is likely to have a child like Saul in their constituency. Every one of us bears a responsibility to do all in our power to find a cure for this dreadful disease. I hope that the Government will confirm that they support research for such so-called rare diseases, and will ensure that public policy supports the medical research most likely to produce a cure for Duchenne muscular dystrophy.

1.15 pm

The Parliamentary Under-Secretary of State for Health (Mr. David Lammy) : I commend my hon. Friend the Member for Cardiff, West (Kevin Brennan) on securing this important debate, which I welcome. No one could fail to be moved by the plight of children who suffer from Duchenne muscular dystrophy. I note the support of my hon. Friends the Members for Norwich, North (Dr. Gibson) and for Rhondda (Mr. Bryant), who are at his side.

My hon. Friend the Member for Cardiff, West mentioned Nick and Janet Catlin's charity, Parent Project UK. In preparing for the debate, I looked at the charity's website and read with great interest and humility about the plight of their son, Saul, and their work to raise awareness of the disease. There was a muscular dystrophy awareness week at the end of September, which I hope was successful in making the public more aware of these distressing diseases. I realise how serious the issue is for families whose children have the genetic defect that causes DMD. I admire Mark and Zoe Thein's effort to raise awareness of DMD by walking to Parliament from Peterborough.

As the hon. Gentleman mentioned, there is a meeting on the subject later today in the House. I am sad to say that my commitments mean that I cannot attend, but my hon. Friend the Member for Edmonton (Mr. Love), the Parliamentary Private Secretary to the Minister of State, Department of Health, my hon. Friend the Member for Redditch (Jacqui Smith), will attend to hear more about the condition first hand.

My hon. Friend the Member for Cardiff, West asked about some of the initiatives under way to help patients with this awful condition. They range from initiatives to look into the cause of the disease, through to the Department's providing much-needed assistance to those who suffer the condition so that they can maintain as good a quality of life as possible.

As my hon. Friend explained, DMD is an inherited, severe and progressive muscle-wasting disease that is estimated to affect around one in every 3,500 boys each year. It is caused by a genetic defect characterised by progressive weakness and degeneration of the skeletal or voluntary muscles that control movement. Most boys with the disease will be in a wheelchair by the age of 10.

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Tragically, there is currently no effective cure or treatment for Duchenne muscular dystrophy. I therefore understand why the families of children with DMD call for a greater research effort to be applied. The main agency through which the Government support medical and clinical research is the Medical Research Council. The MRC is an independent body that receives its grant in aid from the Office of Science and Technology. However, it is a long-standing and important principle of successive Governments that they do not prescribe the detail of how individual research councils should distribute their resources between competing priorities. Researchers and research users should best decide that. I will, however, make sure that, through the offices of the chief medical officer, a copy of the report of the debate is placed before the Medical Research Council. I welcome the fact that my hon. Friend seeks to discuss the condition further.

The Government are committed to investing in genetic research. In 2001–02, the MRC will spend about £2 million on research into Duchenne muscular dystrophy. That is higher than the figure that we gave in April. The MRC is now funding five projects on various aspects of DMD, and I can provide details of them separately if my hon. Friend wishes. Other research projects are being carried out on muscular dystrophy, and on basic underpinning work that could apply to all forms of the disease.

The MRC does not usually earmark funds for particular topics: proposals for research in all areas compete for the available funding. When appropriate, high-quality research in the areas that we are prompting may be given priority in the competition for funds, but research excellence and importance to health will continue to be the primary considerations in funding decisions. In addition, although prevalence of a particular condition is an important factor, the MRC's funding decisions are based largely on scientific opportunity and the likelihood of significant development.

The report from the chief medical officer's expert group, published in 2000, is entitled "Stem cell research: medical progress with responsibility". It recognises that early research on stem cells is exciting and that it provides a real hope of new treatments becoming available in a few years for people with chronic diseases. I welcome my hon. Friend's comments in that regard.

The Government welcome the publication of the report of the House of Lords Health Committee on stem cell research, which endorsed the importance of research using embryonic stem cells. That report also upheld the Government's position that research using cell nuclear replacement—therapeutic cloning—is justified under the existing strict controls operated by the Human Fertilisation and Embryology Authority.

In January, the Secretary of State announced plans for the first national network of genetic knowledge parks. The aim is to put Britain at the leading edge of advances in genetic technology, which could transform treatments and services for NHS patients. We are investing £10 million in the genetics knowledge challenge fund, and the Department of Trade and Industry will contribute a further £5 million. There will be six genetics knowledge parks and two new national genetics reference laboratories. The genetics revolution is already under way. It is changing the world in which

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we live; it holds out the potential for new drugs and therapies, for new means of preventing ill health and for new ways of treating illness.

In time, we should be able to assess an individual's risk of developing not only single-gene disorders such as DMD and cystic fibrosis, but our country's biggest killer diseases—cancer and coronary heart disease—as well as other diseases such as diabetes which limit people's lives.

The potential of genetic research is immense. Although genetics will never lead to a disease-free existence, greater understanding of genetics is one of our best allies in the war against disease. Advances in genetics will lead to a greater understanding of the causes of genetic defects and possible treatments to resolve them. I know that there are exciting developments in America and elsewhere using mice, and these advances have given hope to the parents of boys with DMD. We must recognise that genetic interventions are probably still some years away, although with the amount of research currently being funded worldwide, it is impossible to predict where and when breakthroughs will come. In the meantime, we must try to ensure that those children who suffer from the disease are supported and cared for by the health service to the best of its ability.

An important recent development in the management of long-term conditions has been the recognition that people who live with illnesses such as DMD have considerable knowledge of their condition. That means that NHS health care services need to be reorientated to ensure a more equal partnership between patients and health and social care professionals.

The 1999 document "Saving Lives: Our Healthier Nation", a public health strategy White Paper, proposed a programme to provide NHS-based training in techniques of self-management, allowing people with chronic conditions to develop skills in partnership with their health and social care professionals to manage their condition from day to day. An expert task force with representatives from voluntary, professional and clinical organisations, health service users and carers was established. Its recommendations for an NHS expert patients programme formed the basis of the report "The Expert Patient: A New Approach to Chronic Disease Management for the 21st Century", published in September 2001. Where appropriate, it will also promote partnership between users, carers and care professionals in the development and delivery of training. It will feature a mix of generic and disease-specific training and be piloted between 2002 and 2004 in each primary care trust in England. We expect that it will become integral to the mainstream NHS health care provision between 2004 and 2007.

DMD does not only affect the patient; it has a major impact on the whole family. I do not underestimate the role played by carers and the difficulties that they face in trying to maintain a normal family life. During the past few years, our national carers strategy has achieved much in improving carers' access to breaks, information and support. The Government are increasing the amount of money put aside specifically to support carers in England through the carers grant. In 2002-03, the grant is worth £85 million. By 2005-06, it will more than double to £185 million. The increase in the carers grant

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will ensure that 130,000 more carers benefit from short-term breaks and the extended care that enables them to continue to care.

The Carers and Disabled Children Act 2000 came into force in April 2001. The Act strengthens the rights of carers to an assessment of their needs. It gives local councils more powers directly to support carers by making direct payments to them. It makes provision for carers' services through the carers grant, as well as for flexible breaks through the short-term break voucher scheme, which will be implemented later this year.

During the past decade, the Department of Health has also provided support to the umbrella charity, the Genetic Interest Group, a national alliance of organisations that support children, families and individuals affected by genetic disorders, of which the Duchenne Family Support Group is a member.

In February 2001, the Secretary of State for Health announced the development of a national service framework for long-term conditions. The NSF will focus particularly on the needs of people with neurological conditions and brain and spinal injury. However, the NSF must also tackle some of the generic issues affecting a wide range of people with long-term conditions, and their families and carers. We hope that work to establish standards for neurological conditions will provide patterns of service provision that have wider application and will benefit people with non-neurological conditions.

I hope that my hon. Friend agrees with me that the Government are sympathetic to the needs of patients with Duchenne muscular dystrophy. The initiatives that I described, such as the important research work that is being done, the expert patients programme, the carers' strategy and the national service framework, will help make life better for patients with DMD, and for their families and carers.

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